Development of a Quantitative In Vivo Potency Assay for AVXS-101 Using the Delta 7 Mouse Model of Spinal Muscular Atrophy

Allan Kaspar,Lyndsey Braun,Stephanie Solano,Katherine Nguyen,Elle Lang,Kevin Foust, Guoxiang Chen, Fred Kamal, James L'Italien,Brian K. Kaspar

MOLECULAR THERAPY(2019)

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